May 30, 2013
Researchers at the Perelman School of Medicine, University of Pennsylvania have developed a new gene therapy to thwart a potential influenza pandemic. Specifically, investigators in the Gene Therapy Program, Department of Pathology and Laboratory Medicine, directed by James M. Wilson, MD, PhD, demonstrated that a single dose of an adeno-associated virus (AAV) expressing a broadly neutralizing flu antibody
into the nasal passages of mice and ferrets gives them complete
protection and substantial reductions in flu replication when exposed to
lethal strains of H5N1 and H1N1 flu virus. These strains were isolated from samples associated from historic human pandemics - one from the infamous 1918 flu pandemic and another from 2009.
Wilson, Anna Tretiakova, PhD, Senior Research Scientist, Maria P.
Limberis, PhD, Research Assistant Professor, all from the Penn Gene
Therapy Program, and colleagues published their findings online this
week in Science Translational Medicine ahead of print. In
addition to the Penn scientists, the international effort included
colleagues from the Public Health Agency of Canada, Winnipeg; the
University of Manitoba, Winnipeg; and the University of Pittsburgh.
Tretiakova is also the director of translational research, and Limberis
is the director of animal models core, both with the Gene Therapy
"The experiments described in our paper provide critical
proof-of-concept in animals about a technology platform that can be
deployed in the setting of virtually any pandemic or biological attack
for which a neutralizing antibody exists or can be easily isolated,"
says Wilson. "Further development of this approach for pandemic flu has
taken on more urgency in light of the spreading infection in China of
the lethal bird strain of H7N9 virus in humans."
At the Ready
infections are the seventh leading cause of death in the United States
and result in almost 500,000 deaths worldwide per year, according to the
Centers for Disease Control. The emergence of a new influenza pandemic
remains a threat that could result in a much loss of life and worldwide
There is also interest by the military in developing an off-the-shelf prophylactic vaccine should soldiers be exposed to weaponized strains of infectious agents in biologic warfare.